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Erenumab for Chronic Cluster Headache: A Randomized Clinical Trial
Jasper Mecklenburg1, Charly Gaul2, Mira Fitzek1,3
1Department of Neurology, Charité Universitätsmedizin Berlin, Corporate Member of Free University and Humboldt University of Berlin, Berlin, Germany.
JAMA Network Open
|June 17, 2025
Summary
Erenumab, a CGRP receptor antagonist, did not prove effective in preventing chronic cluster headaches (CCH) in a clinical trial. Further research is needed to understand CGRP
Area of Science:
- Neurology
- Pharmacology
Background:
- Calcitonin gene-related peptide (CGRP) plays a role in cluster headache (CH) pathophysiology.
- Limited prophylactic treatment options exist for chronic cluster headache (CCH).
Purpose of the Study:
- To evaluate if erenumab, a CGRP receptor antagonist, is superior to placebo for CCH prophylaxis.
Main Methods:
- A 12-week, double-blind, placebo-controlled randomized clinical trial (CHERUB01) involving 81 participants with CCH.
- Participants received either erenumab (loading dose 280 mg, then 140 mg) or placebo subcutaneously.
- Primary endpoint: reduction in mean weekly CH attacks over weeks 5 and 6.
Main Results:
- Erenumab did not significantly reduce weekly CH attacks compared to placebo (mean reduction -7.3 vs -5.9 attacks).
- No significant difference in 50% responder rates or Patient Global Impression of Improvement (PGI-I) scores between groups.
- Adverse events were more frequent with erenumab (65.9%) than placebo (42.5%), mostly mild to moderate.
Conclusions:
- CGRP receptor blockade with erenumab was not effective for CCH prophylaxis in this trial.
- The study suggests further investigation into CGRP's role in CCH is warranted.
